New Results From Landmark NURTURE Study Show That Pre-Symptomatic
SMA Patients Treated With SPINRAZA® (nusinersen) Continue to
Demonstrate Sustained Benefit From Treatment
Biogen Inc. (Nasdaq: BIIB) today announced new results
from NURTURE, the longest study of pre-symptomatic patients with
spinal muscular atrophy (SMA) that is transforming expectations of
early treatment with SPINRAZA (nusinersen). In infants genetically
diagnosed with SMA, new data demonstrate that early and sustained
treatment with SPINRAZA for up to 4.8 years enabled unprecedented
survival. Patients continued to maintain and make progressive gains
in motor function compared to the natural course of the disease.
These results are being presented at the virtual Cure SMA Research
& Clinical Care Meeting taking place June 10-12, 2020.
The new data include nearly a year of additional follow-up for
NURTURE study participants. As of February 2020, all patients
treated (n=25; median age of 3.8 years old) were alive and remained
free of permanent ventilation. In the absence of treatment, the
majority of children with SMA Type 1 would, on average, not reach
their second birthday. Additionally, all children who achieved the
motor milestone of being able to walk independently (many within a
normal timeframe) have maintained that ability from the first
occurrence until the last visit.
“The impact of early and sustained SPINRAZA treatment on these
infants and their families is remarkable. I’ve had the privilege to
watch them grow into active young children, many of whom have
experienced progress in motor function consistent with children
their age who do not have SMA,” said Kathryn Swoboda, M.D., the
Katherine B. Sims, M.D., Endowed Chair in Neurogenetics and
Director of the Neurogenetics Program, Massachusetts General
Hospital. “The new results from NURTURE continue to bolster the
substantial benefit of both prompt diagnosis and early and
longer-term treatment with SPINRAZA.”
NURTURE is an ongoing, Phase 2, open-label study of 25
pre-symptomatic patients with the genetic diagnosis of SMA (most
likely to develop SMA Type 1 or 2) who received their first dose of
SPINRAZA before 6 weeks old. The study has been extended by an
additional three years, enabling Biogen to evaluate the longer-term
efficacy and safety of SPINRAZA through 8 years of age and further
understand the impact of early treatment. More information on the
NURTURE study (NCT02386553) is available
on clinicaltrials.gov.
Additional results from the updated interim analysis as of
February 2020 show:
- All study participants who were previously able to walk with
assistance (92 percent) and walk independently (88 percent)
maintained that ability over the 11 months since the last data
cut.1
- Over the 11 months of follow-up, one child gained the ability
to walk with assistance (increasing to 96 percent of all study
participants) and also reached the maximum score on the Children’s
Hospital of Pennsylvania Infant Test of Neuromuscular Disorders
(CHOP-INTEND) scale, increasing the total number of study
participants who achieved the maximum score to 21 of 25 (84
percent).
- Patients with two copies of SMN2 were able to score and advance
on the Hammersmith Functional Motor Scale Expanded scale (HFMSE),
which is atypical to the natural history of the disease.
- SPINRAZA was well-tolerated, with no new safety concerns
identified over the extended follow-up period. No children have
discontinued the study due to adverse events associated with
treatment.
About SPINRAZA® (nusinersen)2-4 SPINRAZA is the
first therapy approved to treat infants, children and adults with
spinal muscular atrophy (SMA) and is approved in more than 50
countries. As of March 31, 2020, more than 10,000 individuals have
been treated with SPINRAZA. It is the only SMA treatment to combine
unsurpassed real-world experience with a robust level of clinical
evidence across a broad spectrum of patient populations.
SMA is a rare, genetic, neuromuscular disease that is
characterized by a loss of motor neurons in the spinal cord and
lower brain stem that can result in severe, progressive muscle
atrophy and weakness. Approximately one in 10,000 live births have
a diagnosis of SMA, and people of all ages are impacted by the
disease. It is a leading genetic cause of infant mortality.
SPINRAZA, a foundation of care in SMA, is an antisense
oligonucleotide (ASO), developed using Ionis Pharmaceuticals’
proprietary technology that is designed to target a root cause of
SMA by increasing the amount of full-length survival motor neuron
(SMN) protein, which is critical to maintaining motor neurons. It
is administered by intrathecal injection into the fluid surrounding
the spinal cord where motor neurons reside to deliver the treatment
where the disease starts.
SPINRAZA currently maintains a robust clinical data set in SMA
based on data from approximately 300 patients across a broad range
of SMA populations demonstrating a favorable benefit:risk profile.
SPINRAZA was evaluated in two randomized, double-blind,
sham-controlled studies of infantile and later-onset SMA (ENDEAR
and CHERISH, respectively) and supported by open-label studies that
include pre-symptomatic infants (NURTURE), individuals with
later-onset SMA (CS2/CS12) and an extension study of individuals
who previously participated in the clinical development program
(SHINE). The most common adverse events observed were respiratory
infection, fever, constipation, headache, vomiting and back pain.
Hypersensitivity, meningitis and hydrocephalus have been observed
in the post-marketing setting. Renal toxicity and coagulation
abnormalities, including acute severe low platelet counts, have
been observed after administration of some ASOs. Laboratory tests
can monitor for these signs.
Biogen licensed the global rights to develop, manufacture and
commercialize SPINRAZA from Ionis Pharmaceuticals, Inc. (Nasdaq:
IONS), a leader in antisense therapeutics. Biogen and Ionis
conducted an innovative clinical development program that moved
SPINRAZA from its first dose in humans in 2011 to its first
regulatory approval in five years.
About BiogenAt Biogen, our mission is clear: we
are pioneers in neuroscience. Biogen discovers, develops and
delivers worldwide innovative therapies for people living with
serious neurological and neurodegenerative diseases as well as
related therapeutic adjacencies. One of the world’s first global
biotechnology companies, Biogen was founded in 1978 by Charles
Weissmann, Heinz Schaller, Kenneth Murray and Nobel Prize winners
Walter Gilbert and Phillip Sharp. Today Biogen has the leading
portfolio of medicines to treat multiple sclerosis, has introduced
the first approved treatment for spinal muscular atrophy,
commercializes biosimilars of advanced biologics and is focused on
advancing research programs in multiple sclerosis and
neuroimmunology, Alzheimer’s disease and dementia, neuromuscular
disorders, movement disorders, ophthalmology, immunology,
neurocognitive disorders, acute neurology and pain.
We routinely post information that may be important to investors
on our website at www.biogen.com. To learn more, please visit
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Biogen Safe Harbor This news release
contains forward-looking statements, including statements made
pursuant to the safe harbor provisions of the Private Securities
Litigation Reform Act of 1995, about the potential benefits, safety
and efficacy of SPINRAZA; the results of certain real-world data;
the identification and treatment of SMA; clinical development
programs, clinical trials and data readouts and presentations; and
the potential benefits and results from early treatment of SMA.
These statements may be identified by words such as “aim,”
“anticipate,” “believe,” “could,” “estimate,” “expect,” “forecast,”
“goal,” “intend,” “may,” “plan,” “possible,” “potential,” “will,”
“would” and other words and terms of similar meaning. You should
not place undue reliance on these statements or the scientific data
presented.
These statements involve risks and uncertainties that could
cause actual results to differ materially from those reflected in
such statements, including without limitation the occurrence of
adverse safety events; risks of unexpected costs, delays or other
unexpected hurdles; failure to protect and enforce our data,
intellectual property and other proprietary rights and
uncertainties relating to intellectual property claims and
challenges; regulatory authorities may require additional
information or further studies; failure to obtain regulatory
approvals in other jurisdictions; product liability claims; third
party collaboration risks; and the direct and indirect impacts of
the ongoing COVID-19 pandemic on our business, results of
operations and financial condition. The foregoing sets forth many,
but not all, of the factors that could cause actual results to
differ from our expectations in any forward-looking statement.
Investors should consider this cautionary statement, as well as the
risk factors identified in our most recent annual or quarterly
report and in other reports we have filed with the U.S. Securities
and Exchange Commission. These statements are based on our current
beliefs and expectations and speak only as of the date of this news
release. We do not undertake any obligation to publicly update any
forward-looking statements, whether as a result of new information,
future developments or otherwise.
References:
- De Vivo DC, Bertini E, Swoboda KJ, et al. Nusinersen initiated
in infants during the presymptomatic stage of spinal muscular
atrophy: Interim efficacy and safety results from the Phase 2
NURTURE study. Neuromuscul Disord. 2019;29(11):842‐856.
doi:10.1016/j.nmd.2019.09.007.
- Based on Commercial Patients, Early Access Patients, and
Clinical Trial Participants as of March 31, 2020.
- Finkel R, Chiriboga C, Vajsar J, et al. Treatment of
infantile-onset spinal muscular atrophy with nusinersen: a phase 2,
open-label, dose-escalation study. Lancet.
2016;388(10063):3017-3026.
- Darras B, Markowitz J, Monani U, De Vivo D. Chapter 8 - Spinal
Muscular Atrophies. In: Vivo BTD, ed. Neuromuscular Disorders of
Infancy, Childhood, and Adolescence (Second Edition). San Diego:
Academic Press; 2015:117-145.
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